Prader-Willi syndrome registry launched

The Global Prader-Willi Syndrome Registry was recently launched by the Foundation for Prader-Willi Research and the National Organization for Rare Disorders to help accelerate research and cures for Prader-Willi syndrome, according to a press release.

The new registry would help create a platform for patients around the world to share their developmental history, medical complications and quality of life with researchers.

Peter L. Saltonstall, president and CEO of the National Organization for Rare Disorders (NORD) said the registry addresses the need for natural history data in order to help researchers understand how the disease develops and progresses over time.

“The Global PWS Registry will provide a complete picture of each patient’s experience with Prader-Willi,” Susan Hedstrom, executive director of the Foundation for Prader-Willi Research (FWPR), said in the release. “We are launching this initiative to help fill the missing link researchers and medical experts need to advance research and get to a cure.”

A custom Facebook Profile Pic creator was developed by the FPWR to help drive awareness and participation.

“Our goal is to enroll as many patients, or their parents or legal guardians, as possible,” Hedstrom said. “The success of the registry is dependent upon community participation.”

Annual electronic surveys will be conducted through the registry to collect patient experience information.

“Another benefit of the Global PWS Registry is its ability to help match participants with potential clinical trials,” Hedstrom said. “If your profile matches the needs of an upcoming clinical trial, you will be notified for the opportunity.”

The program was built using input from patients and patient organizations, researchers and regulators by NORD in an attempt to treat all 7,000 rare diseases.

“We are committed to helping ensure that every rare disease has adequate natural history information and that all rare disease patient organizations have access to the tools they need to advance patient-centered research,” Vincent Tsugranes, director of information technology for NORD, said in the release.

Janet Woodcock, MD, director of the FDA’s Center for Drug Evaluation and Research praised the initiative as a tool “that protects the security and privacy of personal information, while making valuable information available to a research or drug developer interested in creating a new therapy for a rare disease.”

“The Global PWS Registry is the next step in Prader-Willi research and it will advance PWS research faster than ever before,” Hedstrom said.